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ClinicalTrials.gov public records Last synced May 21, 2026, 6:38 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 553 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Lysosomal Storage Diseases Clear all
Not listed No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB
Interventions
Not listed
Lead sponsor
Allievex Corporation
Industry
Eligibility
Up to 18 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Oct 25, 2022 · Synced May 21, 2026, 6:38 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
MPS IVA, Morquio Syndrome
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
1 Year and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 16, 2019 · Synced May 21, 2026, 6:38 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
Interventions
Aldurazyme, placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2005
U.S. locations
8
States / cities
Mobile, Alabama • Atlanta, Georgia • Rochester, New York + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2015 · Synced May 21, 2026, 6:38 PM EDT
Completed Phase 3 Interventional
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 17 Years
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
7
States / cities
San Francisco, California • Aurora, Colorado • Baltimore, Maryland + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced May 21, 2026, 6:38 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Interventions
Laronidase therapy and a stem cell transplant
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 3 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced May 21, 2026, 6:38 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Alzheimer's Disease, Brain Neoplasm, Niemann Pick Disease
Interventions
15 O Water
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
123 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1994 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 21, 2026, 6:38 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Fabry Disease, Proteinuria
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
14 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Nov 18, 2013 · Synced May 21, 2026, 6:38 PM EDT
Terminated Phase 3 Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Tay-Sachs Disease, Sandhoff Disease
Interventions
venglustat GZ402671, placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
2 Years and older
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2026 · Synced May 21, 2026, 6:38 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Gaucher Disease
Interventions
Standard of Care
Other
Lead sponsor
Takeda
Industry
Eligibility
Up to 5 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 28, 2024 · Synced May 21, 2026, 6:38 PM EDT
Completed Phase 4 Interventional Results available
View directory record Official record
Conditions
Gaucher Disease
Interventions
Velaglucerase alfa, Vitamin D
Drug · Dietary Supplement
Lead sponsor
Shire
Industry
Eligibility
18 Years to 70 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
8
States / cities
Beverly Hills, California • Los Angeles, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Jan 31, 2022 · Synced May 21, 2026, 6:38 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced May 21, 2026, 6:38 PM EDT
Approved For Marketing No phase listed Expanded access
View directory record Official record
Conditions
Fabry Disease
Interventions
Pegunigalsidase Alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
U.S. locations
11
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2024 · Synced May 21, 2026, 6:38 PM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced May 21, 2026, 6:38 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Pompe Disease
Interventions
respiratory muscle strength training
Other
Lead sponsor
Duke University
Other
Eligibility
3 Years to 99 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 29, 2023 · Synced May 21, 2026, 6:38 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
Interventions
Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
Diagnostic Test
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced May 21, 2026, 6:38 PM EDT
Active, not recruiting Phase 2 Interventional Results available
View directory record Official record
Conditions
Metachromatic Leukodystrophy (MLD)
Interventions
SHP611
Drug
Lead sponsor
Shire
Industry
Eligibility
6 Months to 72 Months
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
11
States / cities
Torrance, California • Aurora, Colorado • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced May 21, 2026, 6:38 PM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 12, 2019 · Synced May 21, 2026, 6:38 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Morquio Disease, Mucopolysaccharidosis IV
Interventions
Not listed
Lead sponsor
Nadia Ali, PhD
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 3, 2013 · Synced May 21, 2026, 6:38 PM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Fabry Disease
Interventions
Iohexol
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
5 Years to 25 Years · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
5
States / cities
La Jolla, California • Decatur, Georgia • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2020 · Synced May 21, 2026, 6:38 PM EDT
Enrolling by invitation No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis I
Interventions
No Intervention
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced May 21, 2026, 6:38 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Gaucher Disease
Interventions
eliglustat
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 79 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
2
States / cities
Miami, Florida • Knoxville, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 9, 2017 · Synced May 21, 2026, 6:38 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Enchondromatosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced May 21, 2026, 6:38 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced May 21, 2026, 6:38 PM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Sanfilippo Syndrome Type A (MPS IIIA)
Interventions
SOBI003
Drug
Lead sponsor
Swedish Orphan Biovitrum
Industry
Eligibility
18 Months to 78 Months
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
2
States / cities
Oakland, California • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 24, 2022 · Synced May 21, 2026, 6:38 PM EDT